Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

 

Posted on: 6 January 2022, source: Biopharmadive
Avrobio, a Cambridge, Massachusetts-based gene therapy developer, will unexpectedly stop work on one of its most advanced treatments, citing disappointing results from a clinical trial that contrast with earlier, more promising findings. In a statement Tuesday, Avrobio said it would stop recruiting patients into a mid-stage study testing its gene therapy for Fabry disease, a rare inherited disorder, and deprioritize the research program. Monitoring will continue for the 14 Fabry patients who have received Avrobio's therapy so far.

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Posted on: 10 December 2021, source: Pharmaceutical Executive
The creation of the Bespoke Gene Therapy Consortium (BGTC) in the US is welcome news, especially if it achieves its ambition to accelerate the delivery of more gene therapies for rare diseases. The new body is headed and funded by FDA and the National Institutes of Health (NIH), with other stakeholders including small biotechs, major pharma brands, and non-profit organizations.

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Posted on: 30 November 2021, source: Bionews
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to support the process of developing new therapeutics, including tax credits for costs associated with clinical trials, exemptions from certain fees, and marketing exclusivity for seven years if the therapy achieves FDA approval.

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Posted on: 31 October 2021, source: NIH
The National Institutes of Health (NIH) announced the formation of the Bespoke Gene Therapy Consortium (BGTC) on Oct. 27th, 2021. In partnership with FDA, ten pharmaceutical companies, and five non-profits, this coalition is aimed at accelerating development of gene therapies for rare diseases.

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Posted on: 13 October 2021, source: FDA
This guidance provides FDA’s current thinking on determining sameness of human gene therapy products under FDA’s orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity.

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Posted on: 19 September 2021, source: genengnews.com
Astellas Pharma has acknowledged that a fourth boy has died in the troubled Phase I/II trial through which its Astellas Gene Therapies (formerly Audentes Therapeutics) has been evaluating its adeno-associated virus (AAV) gene therapy candidate AT132 in patients with X-linked Myotubular Myopathy (XLMTM). Unlike the other three boys who all died last year in the Phase I/II ASPIRO trial (NCT03199469), the fourth—whose age has not been disclosed—received the lower dose of 1.3×10e14 vg/kg.

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Posted on: 13 September 2021, source: Broad Institute of MIT and Harvard
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene to cells have shown promise in clinical trials for a subset of muscular dystrophies, but have faced challenges. High doses of the gene-carrying virus are needed to reach the muscles throughout the body and the viruses used in these trials often end up in the liver more than in the muscle. This has led to high levels of the virus in the liver, severe adverse side effects, and even death in some trial participants. Researchers at the Broad Institute of MIT and Harvard and Harvard University have developed a new family of adeno-associated viruses (AAVs) - the gene-delivering workhorse of gene therapy - that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle disease.

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